On April 29 & 30 the second 'live' Charlie Consortium meeting took place in the German Mouse Clinic (GMC). The consortium consisting of scientists, clinicians and patient representatives from various countries discussed among others:

• When is Charlie 1.0 a success
• Patients in the Lead; results from the survey posted on the Charlie website
• PDE road to treatment
• GA1 road to treatment
• Manuscripts that will be written
• How to get PDE in New Born Screening
• Podcasts to be created

There was also a separate session with the Scientific Advisory Board where scientific questions were discussed but also the use of anonymous patient survey data and data management.

It appeared all partners are still very enthusiastic to continue in Charlie 1.0 and prepare for a follow-up in Charlie 2.0.

Many thanks to the GMC for hosting this event and the excellent organization.